Medical Safety Follow-up Study for Patients Who Received More Than 28 Days of Total Exposure to BGG492

The purpose of the study was to provide medical follow-up to patients exposed to BGG492 for more than 28 days in study CBGG492A2207 (NCT 01147003) and/or CBGG492A2212 (NCT 01338805). Data from study CBGG492A2216 was used to support the clinical safety assessment of BGG492 regarding the observance of uterine endometrial stromal tumors and adrenal cortical adenomas at least one year after the completion of BGG492 treatment.

• MRI, CT or ultrasound was permitted if MRI was contraindicated (Procedure), also known as Magnetic Resonance Imaging Computed tomography: MRI/CT/ultrasound of abdomen
• Dexamethasone Supression Test (Procedure): Low dose of dexamethasone is administered in the evening; the next morning, a blood sample is collected to measure cortisol
• Sonogram (Procedure), also known as Sonogram ultrasound: Sonogram of the uterus (females only)
• Biopsy (Procedure), also known as Uterine Biopsy: Uterine endometrial biopsy (females only)
• BGG492 (Drug), also known as Selurampanel: No study-drug was administered in this study

Primary outcomes

• Incidence of Adrenal Cortical Adenomas [Minimum of one year after last dose of BGG492 in study BGG492A2207 or BGG492A2212]: Incidence of adrenal cortical adenomas as assessed by non-contrast MRI of the abdomen (CT or ultrasound of the abdomen was permitted if MRI was contraindication)
• Incidence of Uterine Endometrial Stromal Sarcomas [Minimum of one year after last dose of BGG492 in study BGG492A2207 or BGG492A2212]: Incidence of uterine endometrial stromal sarcomas as assessed by sonogram/biopsy (females)

Sex: All, ages: 18 Years to any

Inclusion Criteria:

1. Written informed consent had to be obtained before any assessment was performed;
2. Patients had to be cooperative, willing to participate in the study assessments, and be able to report AEs (adverse events) themselves or have a caregiver who can record and report the events;
3. Total exposure to BGG492 treatment in Study BGG492A2207 and/or BGG492A2212 had to have been greater than 28 days
4. At least 1 year had to have elapsed since the patient received his or her last dose of BGG492.

Exclusion Criteria:

\- There were no exclusion criteria for this study. All patients meeting the inclusion criteria were eligible to participate in the follow-up safety assessments

Participant flow

Outcome results

• Incidence of Adrenal Cortical Adenomas (Primary), Number, Particpants: BGG492 3
• Incidence of Uterine Endometrial Stromal Sarcomas (Primary), Number, Participants: BGG492 0

Adverse events

Serious adverse events by term

Other adverse events by term

• Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals